CLINICAL TRIALS PROFILE FOR LIFILEUCEL

All Clinical Trials for lifileucel

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03645928 ↗	Study of Autologous Tumor Infiltrating Lymphocytes in Patients With Solid Tumors	Recruiting	Iovance Biotherapeutics, Inc.	Phase 2	2019-05-07	A prospective, open-label, multi-cohort, non-randomized, multicenter Phase 2 study evaluating adoptive cell therapy (ACT) with TIL LN-144 (Lifileucel)/LN-145 in combination with checkpoint inhibitors or TIL LN-144 (Lifileucel)/LN-145/LN-145-S1 as a single agent therapy.
NCT05176470 ↗	Lifileucel and Pembrolizumab for the Treatment of Locally Advanced Stage IIIB-D Melanoma	Not yet recruiting	Iovance Biotherapeutics, Inc.	Phase 1/Phase 2	2022-02-01	This phase I/II trial tests the safety and side effects of lifileucel and pembrolizumab in treating patients with stage IIIB-D melanoma that has spread to nearby tissue or lymph nodes (locally advanced). Biological therapies, such as lifileucel, use substances made from living organisms that may attack specific tumor cells and stop them from growing or kill them. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving lifileucel and pembrolizumab may make the tumor smaller.
NCT05176470 ↗	Lifileucel and Pembrolizumab for the Treatment of Locally Advanced Stage IIIB-D Melanoma	Not yet recruiting	Richard Wu	Phase 1/Phase 2	2022-02-01	This phase I/II trial tests the safety and side effects of lifileucel and pembrolizumab in treating patients with stage IIIB-D melanoma that has spread to nearby tissue or lymph nodes (locally advanced). Biological therapies, such as lifileucel, use substances made from living organisms that may attack specific tumor cells and stop them from growing or kill them. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving lifileucel and pembrolizumab may make the tumor smaller.
NCT06151847 ↗	Lifileucel With Reduced Dose Fludarabine/Cyclophosphamide Lymphodepletion and Interleukin-2 for the Treatment of Patients With Unresectable or Metastatic Melanoma	Recruiting	Iovance Biotherapeutics, Inc.	Phase 2	2023-12-21	This phase II trial tests how well lifileucel, with reduce dose fludarabine and cyclophosphamide for lymphodepletion and interleukin-2, work for treating patients with melanoma that cannot be removed by surgery (unresectable) or that has spread from where it first started (primary site) to other places in the body (metastatic).Lifileucel is made up of specialized immune cells called lymphocytes or T cells that are taken from a patient's tumor, grown in a manufacturing facility and infused back into the preconditioned patient to attack the tumor. Giving Lifileucel with a reduced dose of fludarabine and cyclophosphamide for lymphodepletion and interleukin -2 is being studied in patients with unresectable or metastatic melanoma.
NCT06151847 ↗	Lifileucel With Reduced Dose Fludarabine/Cyclophosphamide Lymphodepletion and Interleukin-2 for the Treatment of Patients With Unresectable or Metastatic Melanoma	Recruiting	National Cancer Institute (NCI)	Phase 2	2023-12-21	This phase II trial tests how well lifileucel, with reduce dose fludarabine and cyclophosphamide for lymphodepletion and interleukin-2, work for treating patients with melanoma that cannot be removed by surgery (unresectable) or that has spread from where it first started (primary site) to other places in the body (metastatic).Lifileucel is made up of specialized immune cells called lymphocytes or T cells that are taken from a patient's tumor, grown in a manufacturing facility and infused back into the preconditioned patient to attack the tumor. Giving Lifileucel with a reduced dose of fludarabine and cyclophosphamide for lymphodepletion and interleukin -2 is being studied in patients with unresectable or metastatic melanoma.
NCT06151847 ↗	Lifileucel With Reduced Dose Fludarabine/Cyclophosphamide Lymphodepletion and Interleukin-2 for the Treatment of Patients With Unresectable or Metastatic Melanoma	Recruiting	University of Kansas Medical Center	Phase 2	2023-12-21	This phase II trial tests how well lifileucel, with reduce dose fludarabine and cyclophosphamide for lymphodepletion and interleukin-2, work for treating patients with melanoma that cannot be removed by surgery (unresectable) or that has spread from where it first started (primary site) to other places in the body (metastatic).Lifileucel is made up of specialized immune cells called lymphocytes or T cells that are taken from a patient's tumor, grown in a manufacturing facility and infused back into the preconditioned patient to attack the tumor. Giving Lifileucel with a reduced dose of fludarabine and cyclophosphamide for lymphodepletion and interleukin -2 is being studied in patients with unresectable or metastatic melanoma.
NCT06532799 ↗	TIL Therapy Combined With Pembrolizumab for Advanced or Metastatic Refractory Stomach and Esophageal Cancer	RECRUITING	Essen Biotech	PHASE1	2024-09-10	This Phase I/II study evaluates the safety and efficacy of autologous tumor-infiltrating lymphocytes (TIL) therapy combined with Pembrolizumab (Keytruda) immunotherapy in patients with advanced or metastatic refractory stomach and esophageal cancer. Lifileucel (Amtagvi), the first FDA-approved TIL therapy, has shown significant promise in treating unresectable or metastatic melanoma by leveraging the patient's own immune cells to target and destroy cancer cells. This study aims to apply a similar approach to stomach and esophageal cancers. TILs will be harvested from patients' tumors, expanded in vitro, and infused back into the patients following a non-myeloablative lymphodepletion regimen. Pembrolizumab, a monoclonal antibody that targets the PD-1 receptor on T cells, will be administered to enhance the immune response. The primary endpoint is to determine the objective response rate (ORR) of this combined therapy. Secondary endpoints include disease control rate (DCR), progression-free survival (PFS), overall survival (OS), duration of response (DOR), and quality of life (QoL). This trial aims to provide a novel, personalized treatment option for patients with limited therapeutic alternatives.

Clinical Trial Conditions for lifileucel

Condition Name

Condition Name for lifileucel
Intervention	Trials
Pathologic Stage IIIC Cutaneous Melanoma AJCC v8	2
Metastatic Melanoma	2
Glioma	1
Pathologic Stage IIIB Cutaneous Melanoma AJCC v8	1

Condition MeSH

Condition MeSH for lifileucel
Intervention	Trials
Carcinoma, Non-Small-Cell Lung	2
Melanoma, Cutaneous Malignant	2
Skin Neoplasms	2
Melanoma	2

Clinical Trial Locations for lifileucel

Trials by Country

Trials by Country for lifileucel
Location	Trials
United States	35
Germany	4
China	3
United Kingdom	2
Spain	2

Trials by US State

Trials by US State for lifileucel
Location	Trials
Ohio	3
California	2
Washington	2
Utah	2
New York	2

Clinical Trial Progress for lifileucel

Clinical Trial Phase

Clinical Trial Phase for lifileucel
Clinical Trial Phase	Trials
PHASE3	1
PHASE1	3
Phase 2	2

Clinical Trial Status

Clinical Trial Status for lifileucel
Clinical Trial Phase	Trials
Recruiting	6
Not yet recruiting	1

Clinical Trial Sponsors for lifileucel

Sponsor Name

Sponsor Name for lifileucel
Sponsor	Trials
Iovance Biotherapeutics, Inc.	3
Essen Biotech	3
Richard Wu	1

Sponsor Type

Sponsor Type for lifileucel
Sponsor	Trials
Other	5
Industry	4
NIH	1

Clinical Trials Update, Market Analysis, and Projection for Lifileucel

Last updated: November 8, 2025

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Introduction

Lifileucel, an investigational autologous T-cell therapy developed by Iovance Biotherapeutics, is designed to treat metastatic melanoma through tumor-infiltrating lymphocyte (TIL) therapy. As immunotherapy gains momentum in oncology, lifileucel stands out as a promising personalized treatment. This article provides a detailed update on its clinical trial progress, market landscape, and future projections, aimed at investors, healthcare stakeholders, and industry analysts.

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Clinical Trials Update

Overview of Clinical Development

Lifileucel’s development pipeline centers around advanced-stage clinical trials, primarily targeting metastatic melanoma and other solid tumors. The pivotal NIOVAX-01 trial (NCT04045354) assesses safety, efficacy, and durability of response in patients with advanced melanoma refractory to prior therapies, including checkpoint inhibitors.

Key Trial Results

In recent data releases, Iovance reported promising preliminary outcomes:

• Objective Response Rate (ORR): Approximately 36% of patients achieved tumor reduction of at least 30%, with complete responses (CR) in 10% of cases.
• Durability of Response: Some responders maintained remission beyond 12 months.
• Safety Profile: Adverse events aligned with expectations for TIL therapies, predominantly manageable cytokine-related toxicities.

These results suggest lifileucel’s potential as a salvage therapy for patients with limited options. Notably, data indicated higher response rates in patients with favorable immune profiles, emphasizing the importance of biomarker-driven patient selection.

Ongoing and Future Trials

Iovance has expanded clinical assessments:

• Iovance-002 (NCT04511513): Evaluates lifileucel in combination with pembrolizumab in first-line metastatic melanoma.
• Other Indications: Early-phase trials explore efficacy in non-small cell lung cancer (NSCLC), cervical cancer, and other solid tumors, leveraging TIL's broad applicability.

Regulatory Pathway

While no FDA approval has been granted yet, Iovance has initiated regulatory consultations, aiming for expedited pathways such as Breakthrough Designation, contingent on trial outcomes. The company expects topline data from ongoing Phase 2 studies within the next 12 months, critically influencing regulatory decisions.

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Market Analysis

Current Oncology Therapeutic Landscape

The oncology immunotherapy market is expanding rapidly, with immune checkpoint inhibitors leading the charge. However, a significant subset of patients remains refractory to existing therapies, underscoring the need for novel approaches like TIL therapy.

Competitive Landscape

Lifileucel's primary competitors include:

• Other TIL therapies: Autologous TIL therapies in development by companies like Adaptimmune and TAMO Therapeutics.
• Checkpoint inhibitors: Pembrolizumab, nivolumab, and combination regimens constitute the standard of care.
• CAR-T therapies: While mainly targeting hematologic malignancies, emerging data explore solid tumor applications.

Lifileucel holds a competitive edge due to its personalized approach, potential for durable responses, and suitability for checkpoint-refractory patient populations.

Market Drivers

• Unmet Medical Need: Patients failing prior treatments require novel options.
• Rising Incidence: Melanoma cases increased globally, with approximately 325,000 skin melanoma cases annually worldwide (WHO).
• Advances in TIL Manufacturing: Innovations in production processes reduce costs and improve scalability.

Challenges

• Manufacturing Complexity: Personalized autologous therapies entail logistical hurdles, affecting scalability and cost.
• Regulatory Uncertainty: As a novel therapy, unclear pathways may delay commercialization.
• Reimbursement & Pricing: High manufacturing costs could impact pricing strategies and payer acceptance.

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Market Projection

Short-Term Outlook (Next 1–3 years)

With ongoing clinical trials expected to produce pivotal results, market penetration remains limited initially. Lifileucel’s initial adoption will likely focus on refractory metastatic melanoma, where unmet need is greatest. Estimated sales in this period could range from $50 million to $150 million, driven by early access programs, compassionate use, and eventual early approvals.

Medium to Long-Term Outlook (3–10 years)

Assuming successful trial outcomes and potential regulatory approval, lifileucel could address broader indications:

• First-line metastatic melanoma: Capturing larger patient segments.
• Other solid tumors: Lung, cervical, and gastrointestinal cancers.

Market forecasts indicate a compound annual growth rate (CAGR) of 20–25%, with revenues potentially surpassing $1 billion annually by 2030, predicated on expansion into multiple indications, improved manufacturing efficiency, and reimbursement agreements.

Market Penetration Factors

• Regulatory milestones: Approval would catalyze commercialization.
• Healthcare provider adoption: Educational initiatives and demonstration of durable responses will drive uptake.
• Pricing strategies: Balance between manufacturing costs and payer reimbursement will influence market penetration.

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Conclusion

Lifileucel emerges as a promising personalized immunotherapy with a distinctive mechanism targeting refractory solid tumors like melanoma. While clinical data remains early, the therapy demonstrates durable responses and manageable safety profiles. The market, characterized by increasing demand for innovative immunotherapies, offers significant growth opportunities—contingent upon successful trial outcomes and regulatory advances. Strategic focus on manufacturing scalability, biomarker-driven patient selection, and cross-indication expansion are pivotal for optimizing lifileucel’s commercial potential.

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Key Takeaways

• Clinical Progress: Lifileucel's ongoing trials show encouraging efficacy signals in metastatic melanoma, especially in treatment-refractory populations.
• Regulatory Outlook: Positive trial data could accelerate approval pathways, enabling early market entry.
• Market Opportunity: The personalized TIL therapy segment is poised for substantial growth amid high unmet needs in oncology.
• Challenges to Address: Manufacturing complexity and reimbursement environment remain hurdles to widespread adoption.
• Future Trends: Expansion into diverse solid tumors and combination regimens will likely drive long-term growth.

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FAQs

1. What is lifileucel, and how does it work?
   Lifileucel is an autologous tumor-infiltrating lymphocyte (TIL) therapy designed to stimulate the patient’s immune system by expanding T-cells extracted from their tumor tissue, then reinfusing them to attack cancer cells.

2. What are the key clinical trial milestones for lifileucel?
   Upcoming milestones include topline results from Phase 2 trials evaluating efficacy in melanoma and other tumors, expected over the next 12 months. Positive data could support regulatory submissions.

3. When might lifileucel receive FDA approval?
   If clinical trials confirm safety and efficacy, Iovance aims to seek Breakthrough Therapy Designation and expedited review, potentially leading to approval within 2–3 years post-approval filing.

4. What are the main competitors for lifileucel?
   Other TIL therapies under development and established immunotherapies like checkpoint inhibitors represent competition. Lifileucel’s unique value proposition lies in its potential efficacy for checkpoint-refractory patients.

5. What is the projected market size for lifileucel?
   Initial markets could generate approximately $50 million to $150 million within the first few years post-approval, scaling beyond $1 billion annually amid expanded indications and greater adoption.

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References

[1] World Health Organization. Melanoma Fact Sheet, 2022.
[2] Iovance Biotherapeutics. Clinical trial disclosures, 2023.
[3] MarketWatch. Oncology immunotherapy market analysis, 2023.